BioCryst Pharmaceuticals, Inc. announced FDA approval of ORLADEYO oral pellets for pediatric patients with hereditary angioedema aged 2 to <12.
ORLADEYO is now the first and only targeted oral prophylactic therapy for this patient group, providing a child-friendly method of administration.
The approval was supported by positive interim data from the APeX-P clinical trial, demonstrating early and sustained reductions in monthly attack rates.
FDA Approval
FDA approved ORLADEYO oral pellets for prophylactic therapy in pediatric patients aged 2 to <12 with hereditary angioedema.
Patient Impact
Children with HAE now have an oral prophylactic option, reducing the burden of intravenous or subcutaneous injections.
Clinical Trial
APeX-P trial showed ORLADEYO's safety, tolerability, and efficacy in reducing monthly attack rates in pediatric patients with HAE.
- The approval of ORLADEYO oral pellets marks a significant advancement in providing a convenient and effective treatment option for children with hereditary angioedema.
- This approval expands the age range of patients benefiting from ORLADEYO, addressing the unmet need for younger HAE patients.
- ORLADEYO's unique administration method aims to improve the quality of life for pediatric patients by offering a child-friendly oral formulation.
BioCryst's FDA approval of ORLADEYO oral pellets for pediatric patients with hereditary angioedema aged 2 to <12 represents a milestone in HAE treatment, offering a tailored and convenient prophylactic therapy option for children.